I think $SANA is one of the most hidden gems of all. They have 2 technology platforms with Fusogen and iPSC. Both of these programs could make them one of the biggest potential success stories off all time.
The Fusogen is a delivery vector for in-vivo editing with a much larger carrying capacity. They are targeting in-vivo editing of CAR-T cells. Imagine creating the Autologous CAR-T cells right inside the patient. The amount of cost and time that could be saved.
They can also target the liver and stem cells with this vector for delivery. I think we could see them do in-vivo stem cell corrections like for SCD in the coming years.
Their iPSC programs are amazing with the hypoimmune technology. It makes immune invisible cells by inserting HLA-E into the MHC-I site. Then they upregulate CD47 to avoid macrophages. This has good data in NHP for transplants.
These invisible cells can be used in iPSC to create islet cells for diabetes. They are working on using them for liver, heart and CNS. The potential for these cells across so many indications around regenerative medicine makes this a must own spec stock.
Its still very early, but they have nearly $900 million in cash which can last them a few years. They should be moving programs into the clinic later in 2022 and through 2023. There is plenty of money to last until data.
The NPH data has been amazing so far. It it holds up in the clinic, $SANA stands to redefine what we know about cell therapies. Down here at less then $1.5 billion market cap looks like a gift.
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2/ First demonstration of the survival of allogeneic iPSCs transplanted into an immunocompetent non-human primate model without the need for immune suppression
3/ Transplanting allogeneic cells into a primate without immune suppression represents a key step toward widespread treatment of disease using engineered cells
2/ Designing Hypoimmune Cells
Our goal is to create a universal cell that is able to evade immune detection, regardless of cell type or transplant location.
3/ Our first-generation
technology, which is progressing through late-stage animal confirmatory studies, combines the three gene modifications below to hide these cells from
the host immune system:
This is right from the 10K so Its not my original work, but I don't think I could say it any better.
2/ Background on Fusogens:
Fusogens are a well-studied class of naturally occurring proteins that mediate the trillions of cell-to-cell and intracellular fusion events occurring
in the human body every second.
3/ In 2013, the Nobel Prize in Physiology or Medicine was awarded for the elucidation of the roles of fusogens in
mediating intracellular trafficking in nature. First, fusogens enable recognition of a specific target membrane.
Right now, It is all about the inflation equation. The companies making money right now are trading at bubbly valuations because no one wants to bet on growth companies that promise to make billions 10 years from now. They fear inflation will destroy that promise of growth. $XBI.
As soon as inflation peaks, you will see the rotation begin to move back to growth as value is a bubble. Probably worse then growth was last year. The problem here is the market is sticking with value as it seems the Fed is moving too slowly on getting inflation under control.
This gives you time to sell value, raise cash and slowly nibble away at those growth names while everyone else is scared and hiding in value. I honestly don't know how long it will take to see inflation abate and the switch to flip, but I am willing to buy now and wait.
This is the space that is taking on using stem cells to develop therapies to target diseases.
The cell therapy space is vast, but its my opinion that many of these companies are not even close to being in the right place for the future. Many companies are harvesting cells and editing them. That is just slow and costly.
The future of this space will be to grow trillions of these cells from iPSC cells. This technology offers low cost, high doses and perfect consistency. My top 3 names in this space will be $SANA, $FATE, and $IPSC but I think I will also add is $CRSP.
This is one space where I have a very hard time picking a favorite out of my top 3 of $SDGR, $RXRX and $EXAI. Each of these companies is using technology in a very powerful way to drive drug discovery.
It can take many years and over $1 billion in cost to developing a drug as 90% of new science fails to reach commercial. That is due to most of biotech being trial and error. By using technology, we can lower the cost and improve the chances of success.